LEXINGTON, Mass.–(Company WIRE)–Live performance Prescription drugs, Inc. (NASDAQ: CNCE) these days announced constructive topline outcomes from its a short while ago done Phase 3 medical trial, Thrive-AA1, assessing its oral investigational medication CTP-543 in adult sufferers with average to extreme alopecia areata, an autoimmune disorder that success in patchy or entire scalp hair decline. The most important efficacy endpoint for Prosper-AA1 was the share of clients acquiring an absolute Severity of Alopecia Device (SALT) rating of 20 or significantly less at 7 days 24 of procedure, which was met with statistical significance in both equally the 8 mg twice-daily and 12 mg 2 times-every day dose teams relative to placebo. Treatment with CTP-543 was usually very well tolerated.
The crucial secondary endpoints were being the share of responders on a Hair Gratification Affected person Described Outcome (Professional) scale at Week 24 and the share of clients attaining absolute SALT scores of 20 or considerably less at every single of Weeks 20, 16, 12 and 8. All critical secondary endpoints were fulfilled with statistical importance in both equally dose groups.
“Today marks an critical milestone in advancing new treatment options for alopecia areata, and I’m so joyful to see this kind of optimistic effects from the very first Section 3 demo with CTP-543,” mentioned Brett King, M.D., Department of Dermatology, Yale College Faculty of Drugs and medical investigator of Thrive-AA1. “There is a good need to have for therapies for this complicated ailment, and the outcomes from the Thrive-AA1 trial advise that CTP-543 may likely provide an essential therapy for dealing with alopecia areata.”
“With these powerful Phase 3 knowledge, we believe that CTP-543 has the prospective to be a greatest-in-class treatment for people with alopecia areata, a disease that has extensive been dismissed. We are extremely grateful to the clients and teams of clinical exploration experts who participate in our trials,” stated James V. Cassella, Ph.D., Chief Advancement Officer of Concert Prescribed drugs. “We’re performing to improve the treatment landscape and hope that CTP-543 will be 1 of the initial Food and drug administration-accredited treatment options for this really serious disorder.”
Patients enrolled in Prosper-AA1 were needed to have at least 50 per cent scalp hair reduction thanks to alopecia areata, as calculated by SALT. A SALT score of 100 signifies total scalp hair decline while a rating of represents no scalp hair reduction. The common baseline SALT score across all individuals was close to 85.9 (corresponding to significantly less than 15% normal scalp hair protection).
A statistically important proportion of individuals taken care of with possibly 8 mg 2 times-daily or 12 mg twice-every day of CTP-543 skilled bigger scalp regrowth in contrast to placebo. The proportion of sufferers attaining a SALT rating of 20 or a lot less (which means that 80 p.c or more scalp hair protection was realized) was 41.5 % in the 12 mg two times-daily dose group and 29.6 % in the 8 mg twice-every day dose team, compared to .8 p.c of clients in the placebo group, at the 24-week endpoint. The remedy variation for both equally dose teams of CTP-543 relative to placebo was statistically sizeable (p<0.0001).
The safety profile seen with CTP-543 in THRIVE-AA1 was consistent with previous studies. The most common (≥5%) side effects in any dose group were headache, acne, upper respiratory infection, increased creatine kinase levels, COVID-19 infection and nasopharyngitis. Upper respiratory infections were greater in the placebo group than in either of the CTP-543 dose groups. No pulmonary embolisms or deep vein thromboses were observed in the trial. One patient treated with the 8 mg twice-daily dose and one patient treated with the 12 mg twice-daily dose developed herpes zoster (shingles). Serious adverse events were reported in nine patients, with only one patient (in the 8 mg twice-daily dose group) having events (2) that were assessed as possibly related to treatment. Four patients who reported serious adverse events were in the placebo group.
Concert expects to submit the full results from this study for future scientific publication and presentation. These data, along with data from THRIVE-AA2, a second Phase 3 clinical trial, are intended to form the basis of a New Drug Application (NDA) planned to be submitted to the U.S. Food and Drug Administration (FDA) in the first half of 2023, assuming positive results from THRIVE-AA2. Topline data from THRIVE-AA2 are expected in the third quarter of 2022.
THRIVE-AA1 (NCT04518995) is a randomized, double-blind, placebo-controlled clinical trial in 706 adult patients age 18-65 with moderate to severe alopecia areata at sites in the U.S., Canada and Europe evaluating the regrowth of scalp hair after 24 weeks of dosing using the SALT score. Patients were randomized to receive either 8 mg twice-daily or 12 mg twice-daily of CTP-543 or placebo for 24 weeks. The primary endpoint is the percentage of patients achieving a SALT score ≤ 20 at 24 weeks. All patients who completed 24 weeks of treatment in THRIVE-AA1 had the opportunity to continue in a separate extension study to evaluate long-term safety and efficacy of CTP-543.
About CTP-543 and Alopecia Areata
CTP-543 is an investigational oral selective inhibitor of Janus kinases JAK1 and JAK2. The FDA has granted CTP-543 Breakthrough Therapy designation for the treatment of adult patients with moderate to severe alopecia areata and Fast Track designation for the treatment of alopecia areata.
Alopecia areata is an autoimmune disease in which the immune system attacks hair follicles, resulting in partial or complete loss of hair on the scalp and body. Alopecia areata may affect up to approximately 1.5 million Americans at any given time.1 The scalp is the most commonly affected area, but any hair-bearing site can be affected alone or together with the scalp. Onset of the disease can occur throughout life and affects both women and men. Alopecia areata can be associated with serious psychological consequences, including anxiety and depression. There are currently no drugs approved by the FDA for the treatment of alopecia areata.
The FDA selected alopecia areata as one of eight new disease areas that it focused on under its Patient-Focused Drug Development Initiative (PFDDI) in 2016-2017. The goal of the PFDDI is to bring patient perspectives into an earlier stage of product development. Following the FDA’s Patient-Focused Drug Development meeting held in September 2017 on alopecia areata, the FDA summarized the input shared by patients and patient representatives in a Voice of the Patient report. Additional information on the PFDDI is available online.
Concert Pharmaceuticals is a clinical stage biopharmaceutical company that is developing small molecule drugs that it discovered through the application of its DCE Platform® (deuterated chemical entity platform). Selective incorporation of deuterium into known molecules has the potential, on a case-by-case basis, to provide better pharmacokinetic or metabolic properties, thereby enhancing their clinical safety, tolerability or efficacy. Concert’s lead product candidate is in late-stage development for the treatment of alopecia areata, a serious autoimmune dermatological condition. Concert is also assessing a number of earlier-stage pipeline candidates. For more information please visit www.concertpharma.com or follow us on Twitter at @ConcertPharma or on LinkedIn.
Cautionary Note on Forward Looking Statements
Any statements in this press release about our future expectations, plans and prospects, including, among others, statements about our expectations regarding the development of CTP-543, the potential for CTP-543 to be a best-in-class treatment for the treatment of alopecia areata, the timing of availability of clinical trial data and the timing of regulatory filings, including an NDA for CTP-543, and any other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties inherent in the initiation, timing and design of future clinical trials, the availability and timing of data from ongoing and future clinical trials and the results of such trials, whether preliminary results from a clinical trial will be predictive of the final results of that trial or whether results of early clinical trials will be indicative of the results of later clinical trials, expectations for regulatory approvals and other factors discussed in the “Risk Factors” section of our most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission and in other filings that we make with the Securities and Exchange Commission. In addition, any forward-looking statements included in this press release represent our views only as of the date of this release and should not be relied upon as representing our views as of any subsequent date. We specifically disclaim any obligation to update any forward-looking statements included in this press release.
1 Benigno M. Clinical, Cosmetic and Investigational Dermatology 2020